Ascendis Pharma A/S is set to present two-year data from its Phase 2 ApproaCH trial of TransCon CNP at the International Skeletal Dysplasia Society’s annual meeting in Toronto this week.
The presentation, scheduled for Friday at 1:00 PM during Session 4, will cover Week 104 results from the open-label extension of the trial, which evaluates once-weekly administration of the drug in children with achondroplasia. Ascendis will also present a poster analyzing injection site reactions where indirect treatment comparisons were not feasible.
TransCon CNP, marketed under the brand name YUVIWEL, is a C-type natriuretic peptide prodrug designed to deliver continuous exposure of active CNP to counteract excess FGFR3 signaling in achondroplasia. The U.S. Food and Drug Administration granted approval to YUVIWEL in February 2026 for increasing linear growth in pediatric patients aged two years and older with open epiphyses.
The European Medicines Agency is currently reviewing a marketing authorization application for YUVIWEL, with a regulatory decision expected in the fourth quarter of 2026. Achondroplasia, a rare genetic disorder caused by a variant of the fibroblast growth factor receptor 3 gene, affects more than 250,000 people worldwide and is associated with complications such as spinal anomalies, enlarged brain ventricles, impaired muscle strength, hearing deficits, upper airway obstructions, and chronic pain.
Carlos Bacino, M.D., Professor of Molecular and Human Genetics at Baylor College of Medicine and Texas Children’s Hospital, will deliver the Friday presentation. The ISDS meeting runs from Wednesday to Saturday in Toronto.
